The U.S. Food and Drug Administration has approved the investigational new drug AD-NP1, developed by UCLA scientist Arjun Deb, for use in human clinical trials. The drug is designed to promote tissue repair in the heart and other organs following injury.
Deb’s research identified that after a heart attack, levels of the protein ENPP1 increase in damaged heart tissue. This rise in ENPP1 disrupts energy production and cell function, hindering the healing process. By blocking ENPP1 with the monoclonal antibody AD-NP1, Deb’s team observed improved tissue repair and reduced scar formation in animal studies.
The development of AD-NP1 was funded entirely by public sources including the National Institutes of Health, Department of Defense, and California Institute for Regenerative Medicine. No private investment or corporate involvement was used during its creation.
“This work has been entirely funded by taxpayer dollars, and done entirely within the University of California research ecosystem,” said Deb. “I have not taken a cent from any private donor or company to develop this drug. I hope this will form a model for future drug development at UCLA. This process has advantages of lower costs, potentially shorter development time and the principal investigator being in control of the science and having intellectual freedom with the development of the molecule, which is the most important of all.”
Monoclonal antibodies like AD-NP1 are engineered to mimic natural immune system antibodies but are tailored to target specific proteins—in this case, human ENPP1.
“Much like people eat food to get energy, cells also require energy to multiply and grow and function, and this is more critical when the tissue is injured,” said Deb.
He explained that when pathways generating cellular energy are disrupted by increased ENPP1 expression, cell function declines: “That is what we saw: increased ENPP1 expression interfered with critical pathways that are needed for a cell to derive energy,” he said. He added that using AD-NP1 in animals resulted in stronger heart muscle contractions and prevented heart failure.
Deb believes that because similar metabolic pathways exist across different organs, AD-NP1 could be useful beyond cardiac applications. His approach does not involve stem cells but instead seeks to enhance natural repair mechanisms within tissues.
“Rather, you use the power of the body’s own repair system and optimize it to make it so much better,” said Deb.
If human trials confirm its effectiveness as seen in animals, AD-NP1 could represent a new class of drugs aimed at improving organ recovery after injury.
“Cardiovascular disease is still the leading cause of death in the U.S. and around the world,” said Deb. “All Americans want to lead healthier and longer disease-free lives. It’s a testament to the funding system we have in place in this country that within six or seven years, in an academic lab in a university setting, we have engineered a new drug that potentially could be helpful to many people with heart disease or other forms of organ injury.”
